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Avidity Bio’s RNA Therapy Flashes Early Signs of Treating a Muscular Dystrophy’s Root Cause

Avidity Biosciences’ RNA therapy for facioscapulohumeral muscular dystrophy has preliminary clinical data showing it knocked down expression of a gene that causes this rare disease. More details will be presented at a medical conference this week. By Frank VinluanAn experimental RNA therapy from Avidity Biosciences has early clinical trial results showing it reduced by half the expression of a gene at the root of a rare, inherited form of muscular dystrophy with no FDA-approved therapies. Furthermore, patients in the study are showing signs of regaining muscle function. Based on these initial results announced Wednesday, Avidity said it plans to accelerate the start of additional cohorts in the Phase 1/2 study that could support an application seeking regulatory approval in this disease, facioscapulohumeral muscular dystrophy (FSHD). More details about the data are coming soon. Avidity plans to present the preliminary results during the Annual FSHD Society International Research Congress meeting, which ...
- Source: drugdu
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- June 14, 2024
LucyTx announces additional $12.5m research funding for neurological diseases

Lucy Therapeutics (LucyTx) has announced it has raised $12.5m in additional funding to develop potentially new drugs and advance the company’s research programmes for neurological diseases. The funding, provided by existing investors, Engine Ventures and Safar Partners, with recent participation from the Bill & Melinda Gates Foundation, the Michael J. Fox Foundation and £1.6m from Parkinson’s UK’s Biotech programme, brings the company’s total funding to $36m. The new project aims to develop novel therapies that are based on mitochondrial small-molecule treatments and diagnostic biomarkers for Alzheimer’s (AD) and Parkinson’s disease (PD), as well as continue the development of a novel drug target for Rett syndrome, a rare genetic neurological and developmental disorder that affects brain development. AD and PD are the two most common neurodegenerative diseases worldwide, affecting around 24 million and ten million people, respectively. Using LucyTx’s platform, the team will analyse a variety of disease drivers central to ...
- Source: drugdu
- 78
- June 14, 2024
Iran PHARMA

Organiser：Iran Pharmaceutical Industry Federation Time：October 2nd to 4th, 2024 address：Mashhad Exhibition and Convention Center, Iran Exhibition hall：Mashad Fair Ground Product range: Pharmaceutical machinery: preparation production equipment, pharmaceutical water equipment, traditional Chinese medicine processing equipment, refrigeration equipment, etc Packaging equipment and materials: aluminum-plastic packaging machine, filling machine, tablet press, labeling machine, inkjet printer, anti-counterfeiting printing and packaging and packaging production line, bar code composite technology equipment, adhesive printing and packaging equipment, plastic packaging products, packaging container forming, filling and sealing machine, cartoning packaging machine, bag packaging machine, multi-function bottled packaging machine, glass packaging products and medicinal packaging materials production equipment, etc Laboratory equipment/analysis and testing: laboratory equipment and devices, laboratory consumables, chemical reagents, biochemical/analytical instruments, biotechnology analysis and testing instruments, pharmaceutical testing equipment, food analysis and testing instruments, pharmaceutical industry analysis instruments, etc Fluid equipment: sanitary pumps, valves, fluid engineering, pipes and pipe fittings, microfiltration, ultrafiltration, etc Clean system: clean air ...
- Source: drugdu
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- June 14, 2024
US researchers develop AI liquid biopsy test to help identify lung cancer earlier

Researchers from the Johns Hopkins Kimmel Cancer Center and other institutions have developed and validated a liquid biopsy test using artificial intelligence (AI) to help identify lung cancer earlier. The new study published in Cancer Discovery demonstrated that the new blood test could help accelerate lung cancer screening while reducing death rates. Currently the leading cause of global cancer incidence and death worldwide, lung cancer accounts for an estimated two million diagnoses and 1.8 million deaths annually. For the last five years, researchers have developed a test to detect patterns of DNA fragments found in patients with lung cancer. Participants with and without cancer who met the criteria for low-dose computed tomography (CT) were recruited to receive the blood test to determine which patients were most at risk and would benefit from a follow-up CT screening to help overcome issues regarding appointments, such as the time it takes to arrange ...
- Source: drugdu
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- June 14, 2024
Chiatai Tianqing and China Resources Pharmaceutical Commercial signed a strategic cooperation agreement

On June 7, Chiatai Tianqing Pharmaceutical Group and China Resources Pharmaceutical Commercial signed a strategic cooperation agreement in Tianjin. Based on the principle of resource sharing, complementary advantages and common development, the two parties will carry out in-depth cooperation in the fields of pharmaceutical research and development, production, sales and circulation, and jointly help the sustainable development of China’s pharmaceutical industry. Chiatai Tianqing’s commercial director Qi Tianze and CR Pharmaceuticals Commercial Deputy General Manager Dong Hao signed the agreement on behalf of the two parties. As one of the leading pharmaceutical companies in China, Chiatai Tianqing has always been committed to researching and developing innovative drugs, improving drug quality and meeting patients’ needs. With strong R&D strength and market competitiveness, the company has won wide recognition and trust. As an important pillar industry of China Resources Group, China Resources Pharmaceutical Commercial has a comprehensive pharmaceutical distribution network and market channels, ...
- Source: drugdu
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- June 14, 2024
Innovent Receives Fast Track Designation from the U.S. FDA for IBI343 (TOPO1i anti-CLDN18.2 ADC) as Monotherapy for Advanced Pancreatic Cancer

SAN FRANCISCO and SUZHOU, China, June 13, 2024 /PRNewswire/ — Innovent Biologics, Inc. (“Innovent”) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, cardiovascular and metabolic, autoimmune, ophthalmology and other major diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its TOPO1i anti-CLDN18.2 ADC (R&D code: IBI343), for the treatment of advanced unresectable or metastatic pancreatic ductal adenocarcinoma (PDAC) that has relapsed and/or is refractory to one prior line of therapy. Previously, IBI343 has already received FDA approval of its IND application for the treatment of PDAC. At the ASCO 2024 Annual Meeting, Innovent reported the preliminary Phase 1 results of IBI343 in advanced PDAC patients who have received at least one prior line of treatment. In the 6 mg/kg dose group, among the 10 evaluable PDAC patients with CLDN18.2 1/2/3+≥60%, the overall response ...
- Source: drugdu
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- June 14, 2024
Novel AI-Powered Method for Tissue Analysis Improves Understanding of Disease Pathology

Scientists at Brown University (Providence, RI, USA) and the University of Michigan (Ann Arbor, MI, USA) have created a groundbreaking computational technique to examine complex tissue data, potentially revolutionizing our understanding of diseases and their treatment. The method, known as Integrative and Reference-Informed tissue Segmentation (IRIS), utilizes machine learning and artificial intelligence to provide biomedical researchers with accurate insights into tissue development, disease pathology, and tumor structuring. IRIS employs spatially resolved transcriptomics (SRT) data and incorporates single-cell RNA sequencing data as a reference. This approach allows for the simultaneous examination of multiple tissue layers and accurately identifies different regions with exceptional computational speed and precision. In contrast to traditional methods that offer averaged data from tissue samples, SRT delivers a much more detailed perspective, locating thousands of specific points within a single tissue section. Handling vast and complex datasets has always posed significant challenges, and IRIS addresses these by using ...
- Source: drugdu
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- June 13, 2024
Proteomics Platform Identifies Proteins in Blood to Give Cancer Warning 7 Years before Diagnosis

To improve cancer survival rates, it’s crucial to understand the disease during its initial stages. Research involving data from thousands of cancer patients has uncovered exciting findings about how blood proteins can influence cancer risk, highlighting the need for further investigation to determine which proteins could be effectively utilized for prevention. Researchers have now employed a cutting-edge proteomics platform to identify blood proteins that could potentially provide a seven-year advance warning for various types of cancer. Their study findings underscore the significant potential of proteomics to transform cancer detection, enabling earlier interventions that could lead to improved treatment success and possibly even prevention. In this groundbreaking study recently published in Nature Communications, the researchers used Olink Holding AB’s (Uppsala, Sweden) next-generation Olink Explore proteomics platform to examine blood samples from over 44,000 individuals. They successfully identified 371 proteins associated with 19 different types of cancer, including 107 proteins that could ...
- Source: drugdu
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- June 13, 2024
FDA Approves Regeneron and Sanofi’s Kevzara to Treat Patients with Active Polyarticular Juvenile Idiopathic Arthritis

Don Tracy, Associate Editor Approval for Kevzara was based on controlled studies, pharmacokinetic data from adults with rheumatoid arthritis, and pediatric-specific studies on pharmacokinetics. The FDA has approved Regeneron’s and Sanofi’s approved Kevzara (sarilumab) for the treatment of active polyarticular juvenile idiopathic arthritis (pJIA) in patients weighing over 138 lbs. According to a joint press release, the approval was based on results of controlled studies and pharmacokinetic data from adults with rheumatoid arthritis (RA), as well as pediatric-specific studies on pharmacokinetics, pharmacodynamics, dosing, and safety.1 “Polyarticular juvenile idiopathic arthritis can be a painful disease for children where multiple joints are impacted by this chronic inflammation,” said George D. Yancopoulos, MD, PhD, board co-chair, president, chief scientific officer, Regeneron, in a press release. “Not only are their daily lives impacted, but their futures can be disrupted without adequate treatment. The approval of Kevzara in polyarticular juvenile idiopathic arthritis provides these vulnerable ...
- Source: drugdu
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- June 13, 2024
Ipsen, Genfit Drug That Missed in MASH Wins FDA Approval in a Much Rarer Liver Disease

Iqirvo failed as a treatment for the fatty liver disease MASH, but the drug is now FDA approved in primary biliary cholangitis. An Intercept Pharmaceuticals drug already treats this rare liver disease and Gilead Sciences is poised to compete with its PBC drug approaching an FDA decision this summer. By Frank VinluanA rare liver disease that can progress to organ failure now has a new FDA-approved therapy, a drug from Ipsen that brings a novel approach to treating the chronic condition. The Ipsen drug, elafibranor, treats primary biliary cholangitis (PBC). The regulatory decision announced late Monday makes the drug just the third approved therapy for the chronic liver disorder, but competition in this indication is heating up. Paris-based Ipsen will market its new product under the brand name Iqirvo. PBC is an autoimmune condition in which bile and toxins build up in the liver, leading to inflammation and damage to ...
- Source: drugdu
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- June 13, 2024

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